Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

计划入组 2,250 人 · 首次发布于 2023 年 6 月 18 日

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

计划入组 7 人 · 首次发布于 2026 年 6 月 25 日

Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)

计划入组 6 人 · 首次发布于 2012 年 6 月 1 日

tRNA synthetase candidates

Amino acyl-tRNA synthetases (aTyr Pharma)

N-Carbamoyl-Alanine

C₄H₈N₂O₃

Universitetet i Oslo

Norway

I-SHOU UNIVERSITY

Taiwan

i-SENS, Inc.

South Korea

Ravicti Receives Regulatory Approval in Saudi Arabia, Qatar, and Kuwait for Urea Cycle Disorders

FDA Updates Fludarabine Phosphate Labeling for CLL Treatment

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder